- Launched Exclusive In Vivo Gene Insertion
Collaboration with Novartis to Develop Single ARCUS Nuclease as
Potential One-time Hemoglobinopathy Treatment
- Extended Cash Runway to End of 2024
- Reported Updates Across Allogeneic CAR T
Pipeline; Interim Results from Lead PBCAR0191 Study with CAR T
Relapsed Subjects Who Experienced 100% Response Rate
- Published Compelling Preclinical Research in
Molecular Therapy for ARCUS® In Vivo
Chronic Hepatitis B Program
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage
gene editing company developing ARCUS®-based ex vivo allogeneic CAR
T and in vivo gene editing therapies, today announced financial
results for the second quarter ended June 30, 2022 and provided a
business update.
“This past quarter, we have executed on many important aspects
of our business. From manufacturing optimization to clinical trial
progress to business development and operational advancements, we
are making strides towards delivering on the potential of ARCUS
genome editing for drug development and adding significant cash to
our balance sheet. Most exciting this past quarter, we entered into
a collaboration agreement with Novartis to develop a potential
one-time treatment option for hemoglobinopathies including sickle
cell disease and beta thalassemia using ARCUS for gene insertion.
We are very excited about expanding ARCUS into potential
indications that require more complex edits, such as gene
insertion,” said Michael Amoroso, Chief Executive Officer at
Precision BioSciences.
“For our CAR T programs, Q2 2022 was also an exciting time for
us to share encouraging new interim clinical data for our lead
PBCAR0191 program. We believe the latest update validated the
signal of high response rates to PBCAR0191 observed among CAR T
relapsed patients we reported at the 2021 American Society for
Hematology (ASH) meeting, further supporting our potential path
forward in this patient population. We have recently released
optimized batches of our PBCAR19B clinical trial material and look
forward to commencing dosing in the next cohort of patients this
quarter. In addition, we dosed PBCAR269A in combination with
nirogacestat at Dose Level (DL) 3. We expect to provide additional
updates across our CAR T portfolio by the end of the year,”
concluded Michael Amoroso.
Recent Developments and Upcoming Milestones:
Ex Vivo Allogeneic CAR T Portfolio:
PBCAR0191: PBCAR0191, azercabtagene zapreleucel
(azer-cel), is Precision’s lead investigational anti-CD19
allogeneic CAR T candidate in a Phase 1/2a clinical trial of adult
subjects with relapsed or refractory (R/R) non-Hodgkin lymphoma
(NHL). In June 2022, Precision provided new interim data as of May
31, 2022, including high and durable clinical response rates among
subjects who received a median of five prior lines of therapy.
Evaluable subjects in the latest cohort of the study had the
following results:
-
100% Overall Response Rate (ORR) and 73%
Complete Response (CR) rate
-
50% durable response rate greater than six
months
-
No Grade 3 or greater cytokine release
syndrome (CRS) was observed in either dosing cohort. One Grade 3
immune effector cell-associated neurotoxicity syndrome (ICANS) was
recorded in each cohort that rapidly resolved to Grade 1 within 24
to 48 hours. Two Grade 5 events associated with late occurring
encephalopathy suspected to be related to fludarabine-associated
neurotoxicity occurred. There was no evidence of graft versus host
disease.
In the second half of 2022, Precision plans to continue dosing
subjects with optimized PBCAR0191 CAR T cells in this relapsed
patient population while further reducing its lymphodepletion dose
to standard levels.
PBCAR19B: PBCAR19B is Precision’s second generation,
anti-CD19 targeting allogeneic CAR T candidate designed to evade
immune rejection by host T cell and natural killer (NK) cells with
a single-gene edit to knock-down beta-2 microglobulin and insert an
HLA-E transgene. Manufacturing optimization for PBCAR19B was
implemented in the first quarter of 2022. New clinical trial
material has been released, and the company plans to commence
dosing in the next cohort, DL 2 (flat dose of 540 million cells),
in the third quarter of 2022.
PBCAR269A: PBCAR269A is Precision’s investigational
allogeneic CAR T cell candidate targeting B-cell maturation antigen
(BCMA) for R/R multiple myeloma. Precision is evaluating PBCAR269A
in a Phase 1/2a study in combination with nirogacestat, a gamma
secretase inhibitor (GSI) developed by SpringWorks Therapeutics.
Precision has completed DL2 (2.0 × 106 cells/kg) of PBCAR269A plus
GSI and is initiating the next cohort at DL3 (flat dose of 480 ×106
cells) to further evaluate efficacy. To date, peak expansion rates
observed at DL2 plus the GSI have been equivalent to DL4 (960 × 106
cells flat dose) monotherapy with no dose limiting toxicities
observed.
Precision expects to provide the next update on its clinical ex
vivo allogeneic CAR T programs toward year-end 2022.
In Vivo Gene Editing Portfolio:
Novartis In Vivo Gene Editing Collaboration: In June
2022, Precision announced it had entered into an exclusive
worldwide in vivo gene editing research and development
collaboration and license agreement with Novartis. As part of the
agreement, Precision will develop a single, custom ARCUS nuclease
designed to insert, in vivo, a therapeutic transgene at a “safe
harbor” location in the genome as a potential one-time
transformative treatment option for diseases including certain
hemoglobinopathies such as sickle cell disease and beta
thalassemia. Precision will conduct in vitro characterization, with
Novartis then assuming responsibility for all subsequent research,
development, manufacturing and commercialization activities.
In addition to a $25 million equity investment from Novartis in
Precision’s common stock at $2.01 per share received in June 2022,
Precision received an upfront cash payment of $50 million on July
12, 2022 and is eligible to receive up to an aggregate amount of
approximately $1.4 billion in additional payments for future
milestones. Precision is also eligible to receive certain research
funding and tiered royalties ranging from the mid-single digits to
low-double digits on product sales, should Novartis successfully
commercialize a therapy from the collaboration.
Lilly In Vivo Gene Editing Programs: Precision continues
its in vivo gene editing collaboration with Lilly and is applying
ARCUS nucleases for three initial targets, including Duchenne
muscular dystrophy in muscle, a central nervous system directed
target and a liver directed target.
PBGENE-PH1: Precision has initiated IND-enabling
activities for its PBGENE-PH1 candidate designed to knock out the
HAO1 gene as a potential one-time treatment for primary
hyperoxaluria type 1 (PH1). In the first quarter of 2022, the
Company initiated a non-human primate (NHP) study for PBGENE-PH1
delivered by LNP and is targeting an IND or CTA submission in 2023,
subject to evaluation of data from the ongoing preclinical NHP
study. Preclinical data for Precision’s PBGENE-PH1 was presented at
the 2022 American Society of Gene and Cell Therapy (ASGCT) Annual
Meeting and demonstrated a robust knockdown of the HAO1 protein in
non-human primates following a single administration of an ARCUS
nuclease via AAV.
PBGENE-HBV: Precision’s gene editing program for chronic
Hepatitis B applies ARCUS to knock out persistent covalently closed
circular DNA (cccDNA) and inactivate integrated hepatitis B
genomes, potentially achieving durable HBV S-antigen (HBsAg) loss
and reducing viral persistence. Preclinical data from this program
was presented during the Gene Editing in Cancer and Complex
Diseases oral session at the ASGCT Annual Meeting and within the
same month published online in Molecular Therapy in May 2022. As
reported, ARCUS efficiently targeted and degraded hepatitis B virus
(HBV) cccDNA by 85% and reduced expression of HBsAg by 77% in
HBV-infected primary human hepatocytes (PHH). Importantly, the
optimized specificity of the ARCUS nuclease completely prevented
detectable chromosomal translocations in the PHH model. PBGENE-HBV
candidate is in pre-clinical development and Precision is targeting
an IND/CTA submission in 2024 following completion of pre-clinical
and IND enabling studies.
PBGENE-PCSK9: In 2021, Precision initiated a
collaboration with iECURE, pursuant to which iECURE is expected to
advance Precision’s PBGENE-PCSK9 candidate through preclinical
activities as well as a Phase 1 study in familial
hypercholesterolemia. As of this date, IND enabling activities for
PBGENE-PCSK9 have not been completed. We are in discussions with
iECURE and will provide an update on the program when more
information is available.
Quarter Ended June 30, 2022 Financial
Results:
Cash and Cash Equivalents: As of June 30, 2022, Precision
had approximately $184.1 million in cash and cash equivalents,
including receipts of $25.0 million from Novartis’ equity
investment in the Company and proceeds from the June 2022
underwritten offering of the Company’s common stock, described
below. The Company expects that existing cash and cash equivalents,
including the cash payment of $50.0 million received from Novartis
on July 12, 2022, expected operational receipts, and available
credit will be sufficient to fund its operating expenses and
capital expenditure requirements to the end of 2024.
In June 2022, Precision announced the closing of an underwritten
offering of 35,971,224 shares of its common stock at an offering
price of $1.39 per share, for total net proceeds of approximately
$46.7 million, after deducting underwriting discounts and
commissions and offering expenses payable by the Company.
Revenues: Total revenues for the quarter ended June 30,
2022 were $3.8 million, as compared to $68.8 million for the same
period in 2021. The decrease of $65.0 million in revenue during the
quarter ended June 30, 2022 was primarily the result of the absence
of $62.5 million in revenue recognized under the Servier Agreement
in April 2021 subsequent to full satisfaction of the performance
obligation, a $1.5 million decrease in revenue recognized under the
Lilly Agreement, and a $0.9 million decrease in revenue recognized
from an agriculture partnering collaboration.
Research and Development Expenses: Research and
development expenses were $22.9 million for the quarter ended June
30, 2022, as compared to $37.2 million for the same period in 2021.
The decrease of $14.3 million was primarily due to a decrease of
$11.3 million related to the Servier Program Purchase Agreement, a
decrease of $2.1 million in external development costs associated
with our allogeneic CAR T product candidates, a decrease of $1.3
million in employee-related costs due to reduced headcount, and a
decrease of $1.7 million in clinical manufacturing organization and
research costs related to our preclinical studies. These decreases
were partially offset by a $1.5 million increase in sublicensing
royalty payable to Duke on the Novartis upfront payment.
General and Administrative Expenses: General and
administrative expenses were $10.5 million for the quarter ended
June 30, 2022, as compared to $9.9 million for the same period in
2021. The increase of $0.6 million was primarily due to costs
required to meet our growing infrastructure needs, including
consulting fees and employee-related costs associated with
increased share-based compensation expense.
Net Loss: Net loss was $31.0 million, or $(0.46) per
share (basic and diluted), for the quarter ended June 30, 2022, as
compared to net income of $21.7 million, or $0.38 per share (basic)
and $0.36 per share (diluted), for the same period in 2021.
Corporate:
Executive Leadership: In May 2022, Juli Blanche was
appointed Chief People Officer and a member of the senior
leadership team. Ms. Blanche joined Precision from Bristol-Myers
Squibb where she was Senior Vice President and HR Business Partner,
Global Commercialization. She brings over 25 years of strategic
business partnership and enterprise leadership experience in the
pharmaceutical and financial services industries.
Board of Directors: In May 2022, Melinda Brown was
appointed as a Director on Precision’s Board of Directors and Chair
of the Board’s Audit Committee. Ms. Brown is a financial expert
with proven experience leading accounting, finance and enterprise
risk management teams in large, public companies, including
Tapestry, Inc. and PepsiCo, Inc.
Together, Ms. Blanche and Ms. Brown provide substantial
executive leadership that will be paramount for Precision as the
company embarks upon the next phase of its life cycle.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is a clinical stage biotechnology
company dedicated to improving life (DTIL) with its novel and
proprietary ARCUS® genome editing platform. ARCUS is a highly
precise and versatile genome editing platform that was designed
with therapeutic safety, delivery, and control in mind. Using
ARCUS, the company’s pipeline consists of multiple ex vivo
“off-the-shelf” CAR T immunotherapy clinical candidates and several
in vivo gene editing candidates designed to cure genetic and
infectious diseases where no adequate treatments exist. For more
information about Precision BioSciences, please visit
www.precisionbiosciences.com.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. All statements contained in this press release that do not
relate to matters of historical fact should be considered
forward-looking statements, including, without limitation,
statements regarding the clinical development and expected efficacy
and benefit of our product candidates, benefits of ARCUS and
potential expansion and development using ARCUS, the expected
timing of updates regarding our allogeneic CAR T and in vivo
programs, the expected timing of communications with regulators,
the expected advancement toward and timing of IND and CTA filings,
the ability of our product candidates, if approved, to become
best-in-class or first-in-class, the planned development activities
with our collaboration partners, expectations about our operational
initiatives and business strategy, achieving key milestones and
additional collaborations, and expectations regarding our liquidity
and ability to fund operating expenses and capital expenditures
requirements. In some cases, you can identify forward-looking
statements by terms such as “aim,” “anticipate,” “approach,”
“believe,” “contemplate,” “could,” “estimate,” “expect,” “goal,”
“intend,” “look,” “may,” “mission,” “plan,” “possible,”
“potential,” “predict,” “project,” “should,” “target,” “will,”
“would,” or the negative thereof and similar words and
expressions.
Forward-looking statements are based on management’s current
expectations, beliefs and assumptions and on information currently
available to us. Such statements are subject to a number of known
and unknown risks, uncertainties and assumptions, and actual
results may differ materially from those expressed or implied in
the forward-looking statements due to various important factors,
including, but not limited to: our ability to become profitable;
our ability to procure sufficient funding and requirements under
our current debt instruments and effects of restrictions
thereunder; risks associated with raising additional capital; our
operating expenses and our ability to predict what those expenses
will be; our limited operating history; the success of our programs
and product candidates in which we expend our resources; our
limited ability or inability to assess the safety and efficacy of
our product candidates; our dependence on our ARCUS technology; the
initiation, cost, timing, progress, achievement of milestones and
results of research and development activities, preclinical studies
and clinical trials; public perception about genome editing
technology and its applications; competition in the genome editing,
biopharmaceutical, and biotechnology fields; our or our
collaborators’ ability to identify, develop and commercialize
product candidates; pending and potential liability lawsuits and
penalties against us or our collaborators related to our technology
and our product candidates; the U.S. and foreign regulatory
landscape applicable to our and our collaborators’ development of
product candidates; our or our collaborators’ ability to obtain and
maintain regulatory approval of our product candidates, and any
related restrictions, limitations and/or warnings in the label of
an approved product candidate; our or our collaborators’ ability to
advance product candidates into, and successfully design, implement
and complete, clinical or field trials; potential manufacturing
problems associated with the development or commercialization of
any of our product candidates; our ability to obtain an adequate
supply of T cells from qualified donors; our ability to achieve our
anticipated operating efficiencies at our manufacturing facility;
delays or difficulties in our and our collaborators’ ability to
enroll patients; changes in interim “top-line” and initial data
that we announce or publish; if our product candidates do not work
as intended or cause undesirable side effects; risks associated
with applicable healthcare, data protection, privacy and security
regulations and our compliance therewith; the rate and degree of
market acceptance of any of our product candidates; the success of
our existing collaboration agreements, and our ability to enter
into new collaboration arrangements; our current and future
relationships with and reliance on third parties including
suppliers and manufacturers; our ability to obtain and maintain
intellectual property protection for our technology and any of our
product candidates; potential litigation relating to infringement
or misappropriation of intellectual property rights; our ability to
effectively manage the growth of our operations; our ability to
attract, retain, and motivate key executives and personnel; market
and economic conditions; effects of system failures and security
breaches; effects of natural and manmade disasters, public health
emergencies and other natural catastrophic events; effects of
COVID-19 pandemic and variants thereof, or any pandemic, epidemic
or outbreak of an infectious disease; insurance expenses and
exposure to uninsured liabilities; effects of tax rules; risks
related to ownership of our common stock and other important
factors discussed under the caption “Risk Factors” in our Annual
Report on Form 10-K for the fiscal year ended December 31 2021, as
any such factors may be updated from time to time in our other
filings with the SEC, including, but not limited to, our Quarterly
Report on Form 10-Q for the quarterly period ended June 30, 2022,
to be filed with the SEC, which are accessible on the SEC’s website
at www.sec.gov and the Investors page of our website under SEC
Filings at investor.precisionbiosciences.com.
All forward-looking statements speak only as of the date of this
press release and, except as required by applicable law, we have no
obligation to update or revise any forward-looking statements
contained herein, whether as a result of any new information,
future events, changed circumstances or otherwise.
Precision Biosciences,
Inc.
Condensed Consolidated
Statements of Operations
(In thousands, except share and
per share amounts)
(unaudited)
For the Three Months Ended
June 30,
2022
2021
Revenue
$
3,820
$
68,805
Operating expenses
Research and development
22,936
37,235
General and administrative
10,485
9,938
Total operating expenses
33,421
47,173
Operating (loss) income
(29,601
)
21,632
Other (expense) income:
Loss from equity method investment
(1,448
)
—
Interest expense
(178
)
(24
)
Interest income
192
48
Total other (expense) income, net
(1,434
)
24
Net (loss) income
$
(31,035
)
$
21,656
Net (loss) income per share
Basic
$
(0.46
)
$
0.38
Diluted
$
(0.46
)
$
0.36
Weighted average shares of common stock
outstanding
Basic
67,954,688
57,739,622
Diluted
67,954,688
59,841,638
For the Six Months Ended June
30,
2022
2021
Revenue
$
7,137
$
85,154
Operating expenses
Research and development
42,908
62,828
General and administrative
21,176
19,436
Total operating expenses
64,084
82,264
Operating (loss) income
(56,947
)
2,890
Other (expense) income:
Loss from equity method investment
(2,400
)
—
Interest expense
(220
)
(24
)
Interest income
364
101
Total other (expense) income, net
(2,256
)
77
Net (loss) income
$
(59,203
)
$
2,967
Net (loss) income per share
Basic
$
(0.92
)
$
0.05
Diluted
$
(0.92
)
$
0.05
Weighted average shares of common stock
outstanding
Basic
64,512,356
57,185,402
Diluted
64,512,356
59,647,367
Precision Biosciences,
Inc.
Condensed Consolidated Balance
Sheets Data
(In thousands)
(Unaudited)
June 30, 2022
December 31, 2021
Cash and cash equivalents
$
184,135
$
143,663
Working capital
193,769
125,774
Total assets
297,163
211,498
Total stockholders' equity
$
103,023
$
91,168
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220808005115/en/
Investor Contact: Alex Kelly Chief Financial Officer
Alex.Kelly@precisionbiosciences.com
Media Contact: Maurissa Messier Senior Director,
Corporate Communications
Maurissa.Messier@precisionbiosciences.com
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